Quest Magazine

ABC Television Network Is New Home of MDA Show of Strength Telethon

New Guideline for CMD Diagnosis and Care

This spring, the American Academy of Neurology (AAN) released a guideline for the diagnosis and care of children with congenital muscular dystrophy (CMD), a group of early-onset, genetic disorders that can involve skeletal (including respiratory) muscle weakness, heart problems, brain and eye abnormalities, and contractures (frozen joints).

An early-onset disease with many genetic causes

CMD: Phase 1 Omigapil Trial Remains Open

Santhera Pharmaceuticals continues to recruit participants for a phase 1 trial of omigapil in children and adolescents with the type 1A form of congenital muscular dystrophy (CMD)

MDA to Offer Genetic Testing for Limb-Girdle Muscular Dystrophy

MDA Mourns the Loss of Former National Goodwill Ambassador Mike Neufeldt

FDA OKs Testing of OPMD Drug in US

BioBlast Pharma, based in Tel Aviv, Israel, has been given clearance from the U.S. Food and Drug Administration (FDA) to test its experimental drug Cabaletta in the U.S. in people with oculopharyngeal muscular dystrophy (OPMD).

ALS: Seven Out of 15 Participants in Neuralstem Trial Judged Responders

Maryland-based biotechnology company Neuralstem announced in a March 12, 2014, press release, that a phase 2 trial of its NSI-566 neural stem cells in 15 people with amyotrophic lateral sclerosis (ALS) has shown that the surgical transplantation procedure and the cells

SMA: ISIS-SMNRx To Be Tested in Presymptomatic Infants

Biogen Idec has announced plans to run a phase 2 clinical study of the experimental drug ISIS-SMNRx  in infants with spinal muscular atrophy (SMA)who are less than 6 weeks old and have a genetic diagnosis of the disorder but are not yet showing symptoms. 

MDA Scientific Conference Update: March 14 Morning Sessions

MDA Scientific Conference Update: March 13 Afternoon Sessions

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